Motor neurone disease (MND) is a severely debilitating, incurable and fatal condition that progressively damages parts of the nervous system.
Amyotrophic lateral sclerosis (ALS) is the most common form of MND. MND/ALS is characterised by progressive degeneration of nerve cells in the spinal cord and brain, affecting voluntary control of the arms and legs and eventually leading to difficulty breathing.
MND/ALS has an extremely poor prognosis and has no approved cure or effective treatment to reverse progression. Independent studies have shown that one-third of patients die within 12 months of diagnosis, with an average life expectancy of 27.5 months.
The International Alliance of MND/ALS Associations estimate MND/ALS affects over 350,000 people globally and kills more than 100,000 people annually. While there is currently no cure for MND/ALS, there are limited treatment options. The standard-of-care treatment Riluzole prolongs life by an estimated 3 months.
Monepantel MEND Study
PharmAust is conducting a Phase 1 study of monepantel to treat MND/ALS. The MEND study aims to determine the recommended Phase 2 dose based on safety and preliminary efficacy data. All four planned dose levels have now been completed.
Patients will be eligible to roll-over into an open-label extension (OLE) study that is expected to begin early in 2024. The OLE study will generate valuable safety and efficacy data for a further 12 months.
PharmAust plans to use the data from the Phase 1 MEND study to commence a Phase 2 study in the first half of 2024, and to support applications for an orphan drug designation and investigational new drug application with the US FDA.
PharmAust’s Phase 1 MEND study was supported by a grant of $881,085 from FightMND, Australia’s largest independent not-for-profit organisation for MND/ALS research.